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AveXis announce results from AVXS-101 Phase I trial

05 April 2017

AveXis has released results from the completed Phase I trial of their gene therapy drug for SMA, AVXS-101.

AVXS-101 uses harmless, genetically engineered viruses to increase SMN protein levels, and has recently received “Breakthrough Therapy” status, which could help to speed up the drug approval process (for more information click here).

The completed trial data indicate that AVXS-101 appears to be safe and well tolerated by the young SMA Type 1 patients being treated. Importantly, it also appears to result in improved and sustained muscle function.

The trial included two different treatment strategies. Cohort 1 comprised three patients aged 6-7 months (at the time of treatment) who were given a low virus dose, while Cohort 2 included 12 patients aged 1-8 months and treated with a higher proposed therapeutic dose.

The following highlights from the trial were reported by AveXis, which are accurate as of January 20th, 2017:

  • AVXS-101 continues to be well tolerated by patients, with no new treatment-related safety or tolerability concerns reported since the update in August (for more information click here).
  • All 15 children in the trial remain event-free at 13.6 months of age. An event is defined as the time when ventilation support for breathing is required for at least 16 hours a day for 14 consecutive days, or sadly when a patient dies.
  • The muscle function of AVXS-101-treated patients is continuing to improve, as assessed by a test specifically developed to measure motor function of SMA Type I patients (The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders - CHOP-INTEND).
  • The improvements in motor function are leading to patients consistently achieving and maintaining key developmental motor milestones. In cohort 2, 11/12 patients achieved head control and could sit with assistance, while 5/12 patients could even sit unassisted for 30 seconds or more.

To continue AVXS-101 development, AveXis has recently announced that they are to initiate two pivotal trials of their gene therapy. One will begin in the US in the first half of 2017 (click here for more information) and the other will start in the second half of the year in the European Union (click here for more information).

These new trials will enrol patients with SMA Type 1 and are “single-arm”, meaning that there is no patient group receiving a placebo; rather, motor milestone achievements of patients receiving AVXS-101 will be compared to natural history information from untreated SMA patients.

The two pivotal trials have been designed to provide evidence of drug safety and efficacy in order to determine whether or not to approve AVXS-101 for human use.


Further Information

For additional details on the results from the trial, see the AveXis press release.